Muscular Dystrophy Association - Distal Muscular Dystrophy (DD)https://www.mda.org/taxonomy/term/128
enSimply Stated: What is Muscular Dystrophy?https://www.mda.org/simply-stated-what-muscular-dystrophy
<div class="field field-name-body field-type-text-with-summary field-label-hidden"><div class="field-items"><div class="field-item even" property="content:encoded"><p><a href="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2016/10/20163911/Dystrophy-HP.jpg"><img class="alignright wp-image-3342 size-full" src="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2016/10/20163911/Dystrophy-HP.jpg" alt="muscular dystrophy" width="327" height="230" /></a></p>
<p>Muscular dystrophy is a term that refers to a number of diseases that cause progressive loss of muscle mass resulting in weakness and, sometimes, loss of mobility.</p>
<p>There are many different kinds of muscular dystrophy, each affecting different groups of muscles. In some types of muscular dystrophy, symptoms begin in childhood. In other forms, symptom onset doesn’t occur until adulthood.</p>
<p>Each of the <a href="https://www.mda.org/disease/list">different types of muscular dystrophy</a> is caused by a mutation, or flaw, in a gene that interferes with the production of proteins needed to form and maintain healthy muscle. Such mutations may result in cells manufacturing a defective form of a particular protein, or insufficient (or completely absent) levels of that protein.</p>
<p><b>Muscular dystrophy affects everyday activities</b></p>
<p><figure id="attachment_3355" style="width: 267px" class="wp-caption alignleft"><a href="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2016/10/21111826/Hugo-FF.jpg"><img class="wp-image-3355" src="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2016/10/21111826/Hugo-FF-300x300.jpg" alt="Muscular dystrophy" width="267" height="267" /></a><figcaption class="wp-caption-text">Muscular dystrophy can affect people of all ages, but that won’t stop them from living unlimited</figcaption></figure></p>
<p>Children born with severe forms of muscular dystrophy may never gain the ability to walk or achieve other developmental milestones. Some older children and adults who develop muscular dystrophy may <a href="http://live-strongly-blog.pantheonsite.io/band-basketball-business-oklahoma-man-dmd-lives-unlimited/">eventually lose the ability to walk</a> and will need to use a scooter or wheelchair to help maintain mobility and independence. Some may have trouble breathing, which may require the use of a ventilator to help maintain respiratory function. Others may experience weakness with the muscles involved in swallowing; this can cause nutritional problems that may be alleviated with diet modification and/or the use of a feeding tube.</p>
<p>Depending on the type, muscular dystrophy may involve intellectual disabilities, learning disabilities, eye defects or seizures.</p>
<p>Other complications can include:</p>
<ul><li><b>Contractures. </b><strong>The shortening of muscles or tendons around joints<b> </b></strong>can further limit mobility that already is impaired by muscle weakness.</li>
<li><b>Scoliosis. </b><strong>A curvature in the spine </strong>can occur when weakened muscles are unable to hold the spine straight.</li>
<li><b>Heart problems.</b> Muscular dystrophy can cause changes to the heart muscle that reduce its efficiency.</li>
</ul><p><strong>There is no cure for muscular dystrophy.</strong> But medications and therapy can help manage some symptoms and potentially slow the course of the disease.</p>
<p><figure id="attachment_3354" style="width: 197px" class="wp-caption alignright"><a href="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2016/10/21111706/Ethan-Lybrand-1st-day-of-school.jpg"><img class="wp-image-3354" src="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2016/10/21111706/Ethan-Lybrand-1st-day-of-school-225x300.jpg" alt="Muscular dystrophy" width="197" height="263" /></a><figcaption class="wp-caption-text">There are many different kinds of muscular dystrophy, each affecting different groups of muscles.</figcaption></figure></p>
<p><b>When to seek medical advice</b></p>
<p>Talk with your pediatrician if you notice a lack of muscle tone (called hypotonia) in infants. Additionally, you should let your doctor know if your infant or toddler experiences delays in meeting motor milestones — such as rolling over, sitting up or walking — or fails to meet them at all.</p>
<p>Signs of muscle weakness — such as increased clumsiness and falling — in yourself or your child also may be cause for concern.</p>
<p><em>You can also give our MDA Resource Center a call if you’re looking for one-on-one support at 800-572-1717.</em></p>
<p style="text-align: center;"><strong><br />
Sign up to learn more about how you can help those with muscle debilitating diseases live longer and grow stronger.</strong></p>
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<p>The post <a rel="nofollow" href="https://strongly.mda.org/what-is-muscular-dystrophy/">Simply Stated: What is Muscular Dystrophy?</a> appeared first on <a rel="nofollow" href="https://strongly.mda.org">MDA</a>.</p>
</div></div></div><div class="field field-name-field-strongly-categories field-type-taxonomy-term-reference field-label-above"><div class="field-label">Strongly Categories:&nbsp;</div><div class="field-items"><div class="field-item even"><a href="/taxonomy/term/139" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Research</a></div><div class="field-item odd"><a href="/taxonomy/term/102" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Duchenne Muscular Dystrophy (DMD)</a></div><div class="field-item even"><a href="/taxonomy/term/131" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Limb-Girdle Muscular Dystrophy (LGMD)</a></div><div class="field-item odd"><a href="/taxonomy/term/142" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Oculopharyngeal Muscular Dystrophy (OPMD)</a></div><div class="field-item even"><a href="/taxonomy/term/128" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Distal Muscular Dystrophy (DD)</a></div><div class="field-item odd"><a href="/taxonomy/term/127" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Congenital Muscular Dystrophy (CMD)</a></div><div class="field-item even"><a href="/taxonomy/term/133" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Myotonic Muscular Dystrophy (MMD)</a></div><div class="field-item odd"><a href="/taxonomy/term/140" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Facioscapulohumeral Muscular Dystrophy (FSHD)</a></div></div></div><div class="field field-name-field-strongly-url field-type-text field-label-above"><div class="field-label">Strongly URL:&nbsp;</div><div class="field-items"><div class="field-item even">https://strongly.mda.org/what-is-muscular-dystrophy/</div></div></div>Thu, 27 Jul 2017 19:38:19 +0000Anonymous33711 at https://www.mda.orghttps://www.mda.org/simply-stated-what-muscular-dystrophy#commentsA Mother&#8217;s Powerful Support in the Face of Muscular Dystrophyhttps://www.mda.org/mother%E2%80%99s-powerful-support-face-muscular-dystrophy
<div class="field field-name-body field-type-text-with-summary field-label-hidden"><div class="field-items"><div class="field-item even" property="content:encoded"><p><figure id="attachment_5815" style="width: 300px" class="wp-caption alignright"><img class="wp-image-5815 size-medium" src="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101824/Family-1st-cruise--300x225.jpg" width="300" height="225" srcset="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101824/Family-1st-cruise--300x225.jpg 300w, https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101824/Family-1st-cruise--768x576.jpg 768w, https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101824/Family-1st-cruise--1024x768.jpg 1024w" sizes="(max-width: 300px) 100vw, 300px" /><figcaption class="wp-caption-text">Amy and her siblings and parents on a cruise.</figcaption></figure></p>
<p>Imagine raising four kids. That’s a lot to handle on its own. Now picture that your kids, one by one, started to show symptoms of a mysterious disease that no one could name. How would you react?</p>
<p>These aren’t hypotheticals for me; they describe my life. As each of her children fell ill, my incredible and resilient mother Ann did not face this adversity with fear; she was courageous. As her children got weaker, she got stronger. There is nothing more a mother wants in the world than to make everything better for her children – but this was one thing she couldn’t do. Instead, she chose to shower us with unconditional love, encouragement and support.</p>
<p><figure id="attachment_5820" style="width: 300px" class="wp-caption alignleft"><img class="wp-image-5820 size-medium" src="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101839/Stephs-Wedding-300x235.jpg" width="300" height="235" srcset="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101839/Stephs-Wedding-300x235.jpg 300w, https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101839/Stephs-Wedding-768x601.jpg 768w, https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101839/Stephs-Wedding-1024x801.jpg 1024w" sizes="(max-width: 300px) 100vw, 300px" /><figcaption class="wp-caption-text">Amy’s family at her sister Steph’s wedding.</figcaption></figure></p>
<p>Today, when I look in my mother’s eyes, I feel nothing but peace. We laugh, we talk and we hang out together. I truly believe that we are closer than most families will ever be. I cannot say that I am the best communicator or the best daughter in the world. But what I can say is that I have the best mother that anyone can ask for.</p>
<p><figure id="attachment_5821" style="width: 300px" class="wp-caption alignright"><img class="wp-image-5821 size-medium" src="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101841/Mom-and-Steph-300x225.jpg" width="300" height="225" srcset="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101841/Mom-and-Steph-300x225.jpg 300w, https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101841/Mom-and-Steph.jpg 604w" sizes="(max-width: 300px) 100vw, 300px" /><figcaption class="wp-caption-text">Amy’s mother Ann and sister Steph.</figcaption></figure></p>
<p>At 34, I am the youngest of four siblings living with <a href="https://www.mda.org/disease/distal-muscular-dystrophy/types">GNE Myopathy</a>, an adult onset progressive neuromuscular disease that causes severe muscle weakness in the body sparing the heart, lungs and one’s ability to swallow. For my family, the “adult onset” was earlier than most cases of GNE Myopathy: two of my siblings began having symptoms in their teens; my sister and I started to decline in our early 20s. We didn’t get a correct diagnosis until 2009 when I was 27, more than two decades after my eldest siblings first showed symptoms.</p>
<p><figure id="attachment_5816" style="width: 300px" class="wp-caption alignleft"><img class="wp-image-5816 size-medium" src="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101827/Family-2-300x197.jpg" width="300" height="197" srcset="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101827/Family-2-300x197.jpg 300w, https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101827/Family-2-768x505.jpg 768w, https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101827/Family-2-1024x674.jpg 1024w" sizes="(max-width: 300px) 100vw, 300px" /><figcaption class="wp-caption-text">Family.</figcaption></figure></p>
<p>Although she has never expressed this to me, my mother undoubtedly has struggled with emotional stress about the effects of this hereditary disease. My siblings and I, however, have very clear feelings on these matters: we believe that our parents should not harbor any feelings of guilt, for, without them, we would not have any life at all. Our family got dealt a bad hand in the genetics lottery, but my parents had no way of knowing this before having children.</p>
<p><figure id="attachment_5817" style="width: 300px" class="wp-caption alignright"><img class="wp-image-5817 size-medium" src="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101830/girls-300x225.jpg" width="300" height="225" srcset="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101830/girls-300x225.jpg 300w, https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101830/girls-768x576.jpg 768w, https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101830/girls-1024x768.jpg 1024w" sizes="(max-width: 300px) 100vw, 300px" /><figcaption class="wp-caption-text">Amy, her mother and her sister Linda.</figcaption></figure><br /><figure id="attachment_5819" style="width: 225px" class="wp-caption alignleft"><img class="wp-image-5819 size-medium" src="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101837/Mom-and-dad-225x300.jpg" width="225" height="300" srcset="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101837/Mom-and-dad-225x300.jpg 225w, https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2017/05/13101837/Mom-and-dad-768x1024.jpg 768w" sizes="(max-width: 225px) 100vw, 225px" /><figcaption class="wp-caption-text">Amy’s mother and father.</figcaption></figure></p>
<p>As we enter our 30s and 40s, my siblings and I know that our mother would still do anything for us. Although three of us have moved out and live with our significant others, we are never far apart. My mother’s life still revolves around her children, and she would not have it any other way. She holds all holiday gatherings and parties at her house so that it is accessible for all. She takes over when a personal care assistant calls out or doesn’t show up for a shift. She will take us to the doctor or dentist, babysit her four “grandpuppies,” or pick us up just to hang out together and have fun.</p>
<p>Though our neuromuscular diseases have taken many things from us, they’ve given us a relationship with our mother that is unparalleled. Never one to give up when times got tough, my mother taught us to stick together as a family and support each other through everything. Undoubtedly, she has one of the toughest jobs on earth. But we are the luckiest children. Mom, we love you!</p>
<p style="text-align: center;"><b>Help Adults Like Amy and Her Siblings Live Longer and Grow Stronger!</b></p>
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<p>The post <a rel="nofollow" href="https://strongly.mda.org/a-mothers-powerful-support-in-the-face-of-muscular-dystrophy/">A Mother’s Powerful Support in the Face of Muscular Dystrophy</a> appeared first on <a rel="nofollow" href="https://strongly.mda.org">MDA</a>.</p>
</div></div></div><div class="field field-name-field-strongly-categories field-type-taxonomy-term-reference field-label-above"><div class="field-label">Strongly Categories:&nbsp;</div><div class="field-items"><div class="field-item even"><a href="/taxonomy/term/109" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Personal Stories</a></div><div class="field-item odd"><a href="/taxonomy/term/128" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Distal Muscular Dystrophy (DD)</a></div></div></div><div class="field field-name-field-strongly-url field-type-text field-label-above"><div class="field-label">Strongly URL:&nbsp;</div><div class="field-items"><div class="field-item even">https://strongly.mda.org/a-mothers-powerful-support-in-the-face-of-muscular-dystrophy/</div></div></div>Thu, 27 Jul 2017 19:38:14 +0000Anonymous149326 at https://www.mda.orghttps://www.mda.org/mother%E2%80%99s-powerful-support-face-muscular-dystrophy#commentsNew Research Grants Aim to Help MDA Families Live Unlimitedhttps://www.mda.org/new-research-grants-aim-help-mda-families-live-unlimited
<div class="field field-name-body field-type-text-with-summary field-label-hidden"><div class="field-items"><div class="field-item even" property="content:encoded"><p> </p>
<p><img class="alignright size-full wp-image-2519" src="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2016/08/31103727/GP-head-shot-FINAL-crop-300x2901.jpg" alt="GP-head-shot-FINAL-crop-300x290" width="300" height="290" />This summer MDA challenged our families, friends, sponsors, staff and others around the nation to share their stories about <a href="https://liveunlimited.mda.org/">living life without limits</a> despite the challenges of neuromuscular disease. Some shared moments tied to pursuing an education or career that was thought to be out of reach. Some talked about skydiving, hiking or running a marathon, and others mentioned learning to sing or swim, spending time with family or traveling to faraway places. However #LiveUnlimited moments are defined, MDA is working hard around the clock to make more of them possible.</p>
<p>One of the many ways we’re helping create these precious moments is through our research grants, which fund projects designed to drill down to the bottom of the many diseases covered in our program and work toward finding treatments and cures to slow and stop them.</p>
<p>I’m happy to announce that on Aug. 1 MDA began funding 25 new research, development and research infrastructure grants. This is in addition to 41 such grants awarded earlier this year. Together the two rounds of <a href="https://www.mda.org/research-2016">research awards</a> carry a funding commitment of more than $17 million that is now supporting investigators around the world seeking to advance the state of science and make more #LiveUnlimited moments a reality.</p>
<p><b>Numbers represent proof point of our commitment</b></p>
<p>As we refocus our resources and move closer to our goal of doubling our research investment in drug development and clinical trials testing by 2020, MDA is committed to making treatment options available for all the diseases in our program.</p>
<p>For this latest round of awards, MDA reviewed 226 applications and had sufficient funds to approve funding for approximately 10 percent of them — 25 grants with a total funding commitment of $6.7 million. Here are some highlights:</p>
<ul><li>Laura Ranum, professor of molecular genetics and microbiology at the University of Florida in Gainesville, was awarded an MDA research grant totaling $146,712 over two years to develop an antibody therapy as a potential treatment for <a href="http://www.mda.org/disease/amyotrophic-lateral-sclerosis" target="_blank"><u>amyotrophic lateral sclerosis (ALS)</u></a>.</li>
<li>Pier Lorenzo Puri, associate professor in the development, aging and regeneration program at Sanford Burnham Prebys Medical Discovery Institute in La Jolla, Calif., was awarded an MDA research grant totaling $298,965 over a period of three years to increase understanding about the complexity of cellular interactions that underlie disease progression in <a href="http://www.mda.org/disease/duchenne-muscular-dystrophy" target="_blank"><u>Duchenne muscular dystrophy (DMD)</u></a><i>.</i></li>
<li>Joel Chamberlain, research associate professor at the University of Washington School of Medicine in Seattle, was awarded an MDA research grant totaling $300,000 over a period of three years to increase understanding of the role of DUX4 protein in <a href="http://www.mda.org/disease/facioscapulohumeral-muscular-dystrophy" target="_blank"><u>facioscapulohumeral muscular dystrophy (FSHD)</u></a>.</li>
<li>Marek Napierala, an assistant professor in biochemistry and molecular genetics at the University of Alabama at Birmingham, was awarded an MDA grant totaling $279,518 over a period of three years to investigate a therapeutic strategy for <a href="http://www.mda.org/disease/friedreichs-ataxia" target="_blank"><u>Friedreich’s ataxia (FA)</u></a>.</li>
<li>Madhuri Hegde, associate professor in the department of human genetics at Emory University in Atlanta, was awarded an MDA research infrastructure grant totaling $300,000 over a period of three years to continue groundbreaking work to identify and characterize new gene defects that can cause <a href="http://www.mda.org/disease/limb-girdle-muscular-dystrophy" target="_blank"><u>limb-girdle muscular dystrophy (LGMD)</u></a>.</li>
<li>Marilena D’Aurelio, assistant professor of research in neuroscience at Cornell University in New York, was awarded an MDA research grant totaling $300,000 over a period of three years to examine changes in metabolism that occur in <a href="http://www.mda.org/disease/mitochondrial-myopathies" target="_blank"><u>mitochondrial myopathies (MM)</u></a> and may be used as biomarkers of disease progression.</li>
<li>Lyndsay Murray, a lecturer in anatomy at the University of Edinburgh, in Scotland, United Kingdom, was awarded an MDA research grant totaling $292,174 over a period of three years to investigate how the mechanisms underlying <a href="http://www.mda.org/disease/spinal-muscular-atrophy" target="_blank"><u>spinal muscular atrophy (SMA)</u></a> influence how therapies work at different stages of the disease.</li>
</ul><p>For more information on all the new grants, check out MDA’s <a href="http://www.mda.org/research/grants-at-a-glance" target="_blank"><u>Grants at a Glance</u></a>.</p>
<p>MDA’s current research commitment totals about 150 research projects around the world, each of these a step forward toward treatments and cures.</p>
<p><iframe src="https://www.youtube.com/embed/hb2Pv5zLJIc" width="640" height="360" frameborder="0" allowfullscreen="allowfullscreen"></iframe></p>
<p><b>Grants have impact across disease boundaries</b></p>
<p>At MDA, we know breakthroughs don’t happen in isolation. One of the advantages of being an umbrella organization is the power in the big-picture approach we’re able to take toward finding the breakthroughs that will lead to treatments and cures.</p>
<p>MDA supports the world’s best scientists who are working on projects that will make an impact across the boundaries of the diseases in our program. This means that a single grant could easily advance clinical trial preparedness in both Duchenne and Becker muscular dystrophies; one project may shed light on underlying disease processes in limb-girdle muscular dystrophy, inclusion-body myopathy and myofibrillar myopathies; another may inform therapeutic development for both ALS and spinal muscular atrophy.</p>
<p>As a result of our $1 billion in research investments over the last six decades, MDA’s fingerprints are on nearly every major advance in neuromuscular disease research. With all of our 2016 grant awards, we are continuing to fund the research that will change the neuromuscular disease landscape and make a difference in the lives of our families and loved ones.</p>
<p>Find out how you can fund cures and champion the cause at <a href="http://www.mda.org/"><u>mda.org</u></a>.</p>
<div class="mda-button__container"><a class="button--primary mda-button marigold" href="https://www.mda.org/research-2016" title="Research 2016" target="_blank">Learn more about MDA’s 2016 Research Grants</a></div>
<p>The post <a rel="nofollow" href="https://strongly.mda.org/new-research-grants-aim-to-help-mda-families-live-unlimited/">New Research Grants Aim to Help MDA Families Live Unlimited</a> appeared first on <a rel="nofollow" href="https://strongly.mda.org">MDA</a>.</p>
</div></div></div><div class="field field-name-field-strongly-categories field-type-taxonomy-term-reference field-label-above"><div class="field-label">Strongly Categories:&nbsp;</div><div class="field-items"><div class="field-item even"><a href="/taxonomy/term/139" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Research</a></div><div class="field-item odd"><a href="/taxonomy/term/122" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Grants at a Glance</a></div><div class="field-item even"><a href="/taxonomy/term/112" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Charcot-Marie-Tooth Disease (CMT)</a></div><div class="field-item odd"><a href="/taxonomy/term/102" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Duchenne Muscular Dystrophy (DMD)</a></div><div class="field-item even"><a href="/taxonomy/term/131" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Limb-Girdle Muscular Dystrophy (LGMD)</a></div><div class="field-item odd"><a href="/taxonomy/term/110" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Amyotrophic Lateral Sclerosis (ALS)</a></div><div class="field-item even"><a href="/taxonomy/term/118" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Spinal Muscular Atrophy (SMA)</a></div><div class="field-item odd"><a href="/taxonomy/term/104" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Research Advances</a></div><div class="field-item even"><a href="/taxonomy/term/121" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Grants</a></div><div class="field-item odd"><a href="/taxonomy/term/126" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Friedreich&#039;s Ataxia (FA)</a></div><div class="field-item even"><a href="/taxonomy/term/142" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Oculopharyngeal Muscular Dystrophy (OPMD)</a></div><div class="field-item odd"><a href="/taxonomy/term/130" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Central Core Disease (CCD)</a></div><div class="field-item even"><a href="/taxonomy/term/128" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Distal Muscular Dystrophy (DD)</a></div><div class="field-item odd"><a href="/taxonomy/term/124" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">McArdle Disease</a></div><div class="field-item even"><a href="/taxonomy/term/123" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Spinal-Bulbar Muscular Atrophy (SBMA)</a></div><div class="field-item odd"><a href="/taxonomy/term/127" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Congenital Muscular Dystrophy (CMD)</a></div><div class="field-item even"><a href="/taxonomy/term/132" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Mitochondrial Myopathy (MM)</a></div><div class="field-item odd"><a href="/taxonomy/term/133" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Myotonic Muscular Dystrophy (MMD)</a></div><div class="field-item even"><a href="/taxonomy/term/134" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Myasthenia Gravis (MG)</a></div><div class="field-item odd"><a href="/taxonomy/term/140" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Facioscapulohumeral Muscular Dystrophy (FSHD)</a></div><div class="field-item even"><a href="/taxonomy/term/145" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Inherited and Endocrine Myopathies</a></div></div></div><div class="field field-name-field-strongly-url field-type-text field-label-above"><div class="field-label">Strongly URL:&nbsp;</div><div class="field-items"><div class="field-item even">https://strongly.mda.org/new-research-grants-aim-to-help-mda-families-live-unlimited/</div></div></div>Tue, 13 Jun 2017 16:35:22 +0000Anonymous33971 at https://www.mda.orghttps://www.mda.org/new-research-grants-aim-help-mda-families-live-unlimited#commentsMDA Grants Work to Find Breakthroughs Across Diseaseshttps://www.mda.org/mda-grants-work-find-breakthroughs-across-diseases
<div class="field field-name-body field-type-text-with-summary field-label-hidden"><div class="field-items"><div class="field-item even" property="content:encoded"><p><img class="alignright size-full wp-image-2515" src="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2016/08/31102422/iStock_HowGrantsWork-HP.jpg" alt="iStock_HowGrantsWork HP" width="327" height="230" srcset="https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2016/08/31102422/iStock_HowGrantsWork-HP.jpg 327w, https://s3.amazonaws.com/mda-strongly/wp-content/uploads/2016/08/31102422/iStock_HowGrantsWork-HP-300x211.jpg 300w" sizes="(max-width: 327px) 100vw, 327px" /></p>
<p>Since its inception, <a href="http://www.mda.org/research/what-has-mda-achieved"><u>MDA has invested more than $1 billion</u></a> in neuromuscular disease research to uncover new treatments and cures.</p>
<p>In 2016 alone, MDA <a href="http://mda.org/research-2016">awarded 66 new research</a>, development and research infrastructure grants with a total funding commitment of more than $17 million. These and other MDA grants fund research projects designed to uncover the secrets of the many diseases covered in our program and work toward finding treatments and cures to slow and stop them.</p>
<p>Over the years, we’ve fielded many questions about our research program, including how awards are determined, why there are some diseases that seem to receive more research dollars than others, and why there seems to have been so little progress despite the enormous investment. Here are some of the most frequent questions we receive. Do you have others you are curious about? Let us know in the comments.</p>
<p><b>To whom does MDA award grants?</b></p>
<p><a href="https://www.mda.org/research/research-grants">MDA supports the world’s best scientists</a> who are working on projects that will make an impact across the boundaries of the diseases in our program. Although specific qualifications vary depending on the type of grant, MDA consistently funds the top scientists in the world who are leaders in the neuromuscular disease space.</p>
<p><b>What types of research does MDA support through its grants programs?</b></p>
<ul><li><b>Discovery research</b> includes work to understand the causes of disease, pinpoint the biological pathways involved in disease, identify new drug targets and test new treatment strategies.</li>
<li>MDA’s <b>translational research</b> program is designed to move new drug targets into the clinic as rapidly and efficiently as possible. The program was developed in response to the increasing number of promising therapeutic avenues for neuromuscular diseases that have developed from MDA’s discovery research program.</li>
<li><b>Clinical research</b> supports the continued development of exciting therapies in the pipeline moving into testing in humans. This includes clinical trials, where a new drug or other form of intervention is tested in healthy people (safety trials) or in patients (to see if the intervention alleviates symptoms), but also clinical studies in which researchers are studying the disease itself.</li>
<li>Research to support <b>infrastructure</b> is designed to support the development of tools, techniques and services of need to the neuromuscular research community.</li>
<li>MDA also awards grants to support conferences, meetings and workshops to facilitate the exchange of scientific ideas and crucial information relevant to the diseases under MDA’s umbrella.</li>
</ul><p><b>Where is MDA research happening?</b></p>
<p>MDA funds neuromuscular disease research all around the world. MDA-supported research projects currently are being conducted in 10 countries.</p>
<p><b>When does MDA award grants?</b></p>
<p>Different types of MDA grants may be awarded at different times of the year. Some, including research grants and development grants, are awarded on a regular basis in the winter (February) and summer (August). Other “out of cycle” awards may be made at any time.</p>
<p><b>Why does MDA award grants? </b></p>
<p>Finding treatments and cures is at the heart of MDA’s mission to free individuals — and the families who love them — from the harmful effects of muscular dystrophy, ALS and related diseases. In addition to the work MDA does to provide the best clinical care and support to families living with neuromuscular disease, funding the most promising and innovative research is the top priority.</p>
<p><b>How does MDA decide which grants to fund?</b></p>
<p>The <a href="https://www.mda.org/research/mda-grants-review-process">review process</a> varies depending on the type of grant. For research grants, which make up the majority of MDA’s research funding awards each year, investigators submit grant applications, which are reviewed and rated by MDA’s Research Advisory Committee — a group of approximately 40 of the world’s leading scientists and clinicians who specialize in neuromuscular disease and who serve in volunteer roles for MDA. Grants with the best scores — representing the best and most promising science — are recommended for funding. Approval comes from MDA’s Board of Directors.</p>
<p><b>How many diseases do MDA grants cover?</b></p>
<p>The number of diseases that are the focus of MDA-supported research varies by grant cycle, but many of the research projects we fund have application across the broad spectrum of diseases we cover. MDA is committed to making treatment options available for all the diseases in our program; One of the advantages of being an umbrella organization is the power in the big-picture approach we’re able to take toward finding the medical and scientific breakthroughs that will lead to treatments and cures.</p>
<p><b>Why aren’t you funding research for my disease?</b></p>
<p>MDA-funded research makes an impact across disease boundaries, so although a particular research project may focus on a disease, or group of diseases, the work may have implications that ripple across the neuromuscular disease landscape — this is why MDA’s broad coverage of diseases is so powerful.</p>
<p><b>When you fund research in one disease, aren’t you taking away funding from research into another disease?</b></p>
<p>MDA research dollars aren’t allotted to particular diseases, so funding to support research in one disease area doesn’t “take away” from any other. MDA simply funds the best scientists and the best science out there as we work to find treatments and cures for all the diseases in our program.</p>
<p><strong>Why haven’t we found a cure yet?</strong></p>
<p>There are many neuromuscular diseases, and for some diseases there are even different types. Because there are so many, we will have to find <a href="https://www.mda.org/research/creating-a-new-therapy">many cures</a> — not just one. We’re going to have to treat the different diseases and disease types in many different ways. We can always use more funding to speed the search for cures, as it will allow us to fund more people to work on the various problems. But funding isn’t everything. We also are limited by the speed at which science advances and how our knowledge about the different diseases moves forward.</p>
<p><strong>Why isn’t there more research going into my disease? </strong></p>
<p>In order for research to be conducted for a specific disease, there must be funding available and there must be researchers who are interested in working in the disease area. Both can be positively affected through greater public awareness. MDA is working to raise awareness, attract researchers and accelerate treatments and cures for all the diseases in its program.</p>
<p><strong>How can we encourage more researchers to devote themselves to the study of my disease?</strong></p>
<p>MDA encourages bright, young investigators to pursue careers in the neuromuscular disease research field through grants that are specifically tailored to help launch these young researchers’ scientific programs. As their science advances, many recipients of these development grants apply for and receive research support through MDA’s research grant program. Many have also become members of MDA’s Research Advisory Committee.</p>
<p><strong>What <a href="https://www.mda.org/research/what-has-mda-achieved">successes </a>have come from the MDA research program? </strong></p>
<p>MDA has been involved in research into basic muscle and nerve biology since 1950, when virtually nothing was known about how muscles were formed or functioned, even in non-diseased tissues. Since then, thousands of scientific papers have been published explaining how the tissues work and what goes wrong in neuromuscular disease. MDA funding supported the research that led to the discovery of the genetic causes of dozens of diseases, starting with the discovery of the dystrophin gene in 1986. Without this knowledge, there was no hope for curing diseases.</p>
<p>MDA’s research program has been so successful over the years that MDA started the translational research program in 2004 to help accelerate therapy development based on these results. This program attracted so much interest that it was expanded in 2009 by the formation of <a href="http://www.mda.org/research/mda-venture-philanthropy"><u>MDA Venture Philanthropy (MVP)</u></a>, which operates with a more venture capital-like approach. MVP is exclusively focused on the funding and commercialization of treatments and cures for neuromuscular diseases, and hopes to increase the speed of moving drugs through clinical development.</p>
<p>MDA’s fingerprints are on nearly every major advance in neuromuscular disease research, with MDA-sponsored research having resulted in breakthroughs for treating diseases, and in increasing survival and quality of life.</p>
<p><strong>If MDA did not sponsor this research, what would the <a href="https://www.mda.org/research/state-of-the-science">state of science</a> be?</strong></p>
<p>MDA provides critical funding at a time when many other sources of funding have dried up or become more difficult to obtain. We fund not only the best science and the world’s best researchers, but also the most promising emerging investigators who will make the breakthroughs of tomorrow. MDA funding has kept both the drug development and researcher pipelines moving. Without it, many of the promising therapies in development today may never have gotten off the ground.</p>
<div class="mda-button__container"><a class="button--primary mda-button marigold" href="https://www.mda.org/research-2016" title="Research 2016" target="_blank">Learn more about MDA’s 2016 Research Grants</a></div>
<p> </p>
<p>The post <a rel="nofollow" href="https://strongly.mda.org/mda-grants-work-to-find-breakthroughs-across-diseases/">MDA Grants Work to Find Breakthroughs Across Diseases</a> appeared first on <a rel="nofollow" href="https://strongly.mda.org">MDA</a>.</p>
</div></div></div><div class="field field-name-field-strongly-categories field-type-taxonomy-term-reference field-label-above"><div class="field-label">Strongly Categories:&nbsp;</div><div class="field-items"><div class="field-item even"><a href="/taxonomy/term/139" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Research</a></div><div class="field-item odd"><a href="/taxonomy/term/112" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Charcot-Marie-Tooth Disease (CMT)</a></div><div class="field-item even"><a href="/taxonomy/term/102" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Duchenne Muscular Dystrophy (DMD)</a></div><div class="field-item odd"><a href="/taxonomy/term/131" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Limb-Girdle Muscular Dystrophy (LGMD)</a></div><div class="field-item even"><a href="/taxonomy/term/110" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Amyotrophic Lateral Sclerosis (ALS)</a></div><div class="field-item odd"><a href="/taxonomy/term/118" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Spinal Muscular Atrophy (SMA)</a></div><div class="field-item even"><a href="/taxonomy/term/104" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Research Advances</a></div><div class="field-item odd"><a href="/taxonomy/term/121" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Grants</a></div><div class="field-item even"><a href="/taxonomy/term/126" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Friedreich&#039;s Ataxia (FA)</a></div><div class="field-item odd"><a href="/taxonomy/term/142" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Oculopharyngeal Muscular Dystrophy (OPMD)</a></div><div class="field-item even"><a href="/taxonomy/term/130" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Central Core Disease (CCD)</a></div><div class="field-item odd"><a href="/taxonomy/term/128" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Distal Muscular Dystrophy (DD)</a></div><div class="field-item even"><a href="/taxonomy/term/124" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">McArdle Disease</a></div><div class="field-item odd"><a href="/taxonomy/term/123" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Spinal-Bulbar Muscular Atrophy (SBMA)</a></div><div class="field-item even"><a href="/taxonomy/term/127" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Congenital Muscular Dystrophy (CMD)</a></div><div class="field-item odd"><a href="/taxonomy/term/132" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Mitochondrial Myopathy (MM)</a></div><div class="field-item even"><a href="/taxonomy/term/133" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Myotonic Muscular Dystrophy (MMD)</a></div><div class="field-item odd"><a href="/taxonomy/term/135" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Dejerine-Sottas Disease (DS)</a></div><div class="field-item even"><a href="/taxonomy/term/134" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Myasthenia Gravis (MG)</a></div><div class="field-item odd"><a href="/taxonomy/term/140" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Facioscapulohumeral Muscular Dystrophy (FSHD)</a></div><div class="field-item even"><a href="/taxonomy/term/145" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Inherited and Endocrine Myopathies</a></div></div></div><div class="field field-name-field-strongly-url field-type-text field-label-above"><div class="field-label">Strongly URL:&nbsp;</div><div class="field-items"><div class="field-item even">https://strongly.mda.org/mda-grants-work-to-find-breakthroughs-across-diseases/</div></div></div>Tue, 13 Jun 2017 16:35:21 +0000Anonymous33966 at https://www.mda.orghttps://www.mda.org/mda-grants-work-find-breakthroughs-across-diseases#commentsDD &#8211; Aikaterini Kontrogianni-Konstantopoulos, Ph.D.https://www.mda.org/dd-%E2%80%93-aikaterini-kontrogianni-konstantopoulos-phd-0
<div class="field field-name-body field-type-text-with-summary field-label-hidden"><div class="field-items"><div class="field-item even" property="content:encoded"><p><figure id="attachment_83" style="width: 360px" class="wp-caption alignleft"><img class="wp-image-83 size-full" src="https://strongly.mda.org/wp-content/uploads/2015/12/DD-Aikaterini-Kontrogianni-Konstantopoulos-Ph.D..jpg" alt="Microsoft Word - MDAGrantPublicity.docx" width="360" height="220" srcset="https://strongly.mda.org/assets/uploads/2015/12/DD-Aikaterini-Kontrogianni-Konstantopoulos-Ph.D..jpg 360w, https://strongly.mda.org/assets/uploads/2015/12/DD-Aikaterini-Kontrogianni-Konstantopoulos-Ph.D.-300x183.jpg 300w" sizes="(max-width: 360px) 100vw, 360px" /><figcaption class="wp-caption-text">Aikaterini Kontrogianni-Konstantopoulos is working to deciper how genetic mutations alter the ability of the MyBP-C family of proteins to regulate muscle contractility during disease development.</figcaption></figure></p>
<p>Aikaterini Kontrogianni-Konstantopoulos, an associate professor in the department of biochemistry and molecular biology at the University of Maryland School of Medicine in Baltimore, was awarded an MDA research grant totaling $300,000 over three years to study how the Myosin Binding Protein-C family of proteins may regulate contractile function of skeletal muscle in <a href="http://www.mda.org/disease/distal-muscular-dystrophy/overview">distal muscular dystrophy (DD)</a>. Kontrogianni-Konstantopoulos’ work will shed light on how mutations in this protein lead to disease and may have clinical relevance not only for distal arthrogryposis, but also for other muscular dystrophies as well.</p>
<p><em>Funding for this MDA research grant began Aug. 1, 2015.</em></p>
<p>The post <a rel="nofollow" href="https://strongly.mda.org/dd-aikaterini-kontrogianni-konstantopoulos-ph-d/">DD – Aikaterini Kontrogianni-Konstantopoulos, Ph.D.</a> appeared first on <a rel="nofollow" href="https://strongly.mda.org">MDA</a>.</p>
</div></div></div><div class="field field-name-field-strongly-categories field-type-taxonomy-term-reference field-label-above"><div class="field-label">Strongly Categories:&nbsp;</div><div class="field-items"><div class="field-item even"><a href="/taxonomy/term/100" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Latest from the Lab</a></div><div class="field-item odd"><a href="/taxonomy/term/122" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Grants at a Glance</a></div><div class="field-item even"><a href="/taxonomy/term/104" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Research Advances</a></div><div class="field-item odd"><a href="/taxonomy/term/121" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Grants</a></div><div class="field-item even"><a href="/taxonomy/term/128" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Distal Muscular Dystrophy (DD)</a></div></div></div><div class="field field-name-field-strongly-url field-type-text field-label-above"><div class="field-label">Strongly URL:&nbsp;</div><div class="field-items"><div class="field-item even">https://strongly.mda.org/dd-aikaterini-kontrogianni-konstantopoulos-ph-d/</div></div></div>Wed, 22 Feb 2017 06:08:20 +0000Anonymous34801 at https://www.mda.orghttps://www.mda.org/dd-%E2%80%93-aikaterini-kontrogianni-konstantopoulos-phd-0#commentsDiseases - Distal Myopathies - Top Levelhttps://www.mda.org/disease/distal-myopathies
<div class="field field-name-body field-type-text-with-summary field-label-hidden"><div class="field-items"><div class="field-item even" property="content:encoded"><h3><img alt="" src="/sites/default/files/Overview-body_distalMD.jpg" style="float:right; height:720px; width:300px" />What is distal muscular dystrophy (DD)?</h3>
<p>First described in 1902, DD is a class of muscular dystrophies that primarily affect distal muscles, which are those of the lower arms, hands, lower legs and feet. Muscular dystrophies in general are a group of genetic, degenerative diseases primarily affecting voluntary muscles.</p>
<p><a href="/disease/distal-muscular-dystrophy/types" target="_self">Types of distal muscular dystrophy</a> include: distal myopathy with vocal cord and pharyngeal weakness; Finnish (tibial) distal myopathy; Gowers-Laing distal myopathy; hereditary inclusion-body myositis type 1; Miyoshi distal myopathy; Nonaka distal myopathy; Welander’s distal myopathy; and ZASP-related myopathy.</p>
<h3>What are the symptoms of DD?</h3>
<p>Distal muscular dystrophy can lead to weakness and wasting of muscles of the hands, forearms and lower legs. For more, see <a href="/disease/distal-muscular-dystrophy/signs-and-symptoms" target="_self">Signs and Symptoms</a>.</p>
<h3>What causes DD?</h3>
<p>DD is caused by a mutation in any of at least eight genes that affect proteins necessary to the function of muscles. It can be inherited in an autosomal dominant or recessive pattern. For more, see <a href="/disease/distal-muscular-dystrophy/causes-inheritance" target="_self">Causes/Inheritance</a>.</p>
<h3>What is the progression of DD?</h3>
<p>DD begins in either childhood or adulthood, and is slowly progressive. It doesn't affect the intellect and is not considered life-threatening.</p>
<h3>What is the status of research in DD?</h3>
<p>Recent MDA-supported <a href="/disease/distal-muscular-dystrophy/research" target="_self">research in distal muscular dystrophy</a> has concentrated on understanding how the gene defects that cause this diverse group of diseases affect the proteins made from these genes, and in turn how these protein abnormalities affect muscle tissue.</p>
</div></div></div><div class="field field-name-field-headline-page-title field-type-text field-label-above"><div class="field-label">Headline / Page Title (Purple Box):&nbsp;</div><div class="field-items"><div class="field-item even">Distal Myopathies</div></div></div><div class="field field-name-field-optional-headline field-type-text field-label-above"><div class="field-label">Page Headline:&nbsp;</div><div class="field-items"><div class="field-item even">Overview</div></div></div><div class="field field-name-field-blog-disease-news-tag field-type-taxonomy-term-reference field-label-above"><div class="field-label">Blog Disease News Tag:&nbsp;</div><div class="field-items"><div class="field-item even"><a href="/taxonomy/term/128" typeof="skos:Concept" property="rdfs:label skos:prefLabel" datatype="">Distal Muscular Dystrophy (DD)</a></div></div></div>Fri, 18 Dec 2015 22:05:10 +0000studiog308 at https://www.mda.orghttps://www.mda.org/disease/distal-myopathies#comments